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NCT01390077 · ClinicalTrials.gov registry record · Phase 2
Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria
A Phase 2 study, sponsored by University of California, San Diego.
- Completed
- Registry status
- Phase 2
- Development phase
- 8
- Enrollment target
NCT01390077: Completed Phase 2 study, sponsored by University of California, San Diego.
NCT01390077 is a Phase 2 study that has completed, run by University of California, San Diego. The registered enrollment target is 8 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (94% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01390077, a Phase 2 study, has completed, sponsored by University of California, San Diego.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 8 participants
- Enrollment target
Study Summary
Nitisinone is a potent inhibitor of the enzyme that catalyzes the formation of homogentisic acid, and should be an even more logical treatment for alkaptonuria than for tyrosinemia, for which it has been approved by the FDA.The objective of this research is to explore reported age related differences in toxicity of nitisinone and its pharmacokinetic underpinnings and to develop an optimal therapeutic requirement for a targeted population of presymptomatic patients. The additional effect of mixtures of amino acids excluding tyrosine will be explored to take advantage of protein synthesis to avoid elevations of tyrosine that would otherwise limit the optimal dosage of nitisinone. The study is designed to treat patients and find the optimal dosage of nitisinone to obtain maximal reduction in levels of homogentisic acid and maintain safe levels of tyrosine. The long term objective in the target population of pre-symptomatic patients is the prevention of the characteristic effects on joint cartilage and tendons.
Primary Outcome
Urine homogentisic acid (umol/mmol creatinine)
Interventions
- DRUG Nitisinone
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2011-01 |
| Est. Completion | 2016-07 |
| Phase | Phase 2 |
What the finished NCT01390077 record still lists
NCT01390077 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (94% lower).
The record links to 0 conditions, and to 1 intervention - of which Nitisinone is the first listed.
NCT01390077 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01390077 about?
NCT01390077 is a clinical study titled "Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria". Nitisinone is a potent inhibitor of the enzyme that catalyzes the formation of homogentisic acid, and should be an even more logical treatment for alkaptonuria than for tyrosinemia, for which it has been approved by the FDA.The objective of this research is to explore reported age related difference...
What is the current status of trial NCT01390077?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 8 participants. The study started on 2011-01. Estimated completion is 2016-07.
What interventions are being tested in trial NCT01390077?
The interventions under investigation include: Nitisinone (DRUG).
Who is sponsoring clinical trial NCT01390077?
This trial is sponsored by University of California, San Diego, which has 775 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01390077's enrollment target sits among peer trials
8 1990th of 2000 higher than 5 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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